Ocular gene therapy strategies

Despite the optimistic findings of recent LCA2 studies that resulted in the approval of Luxturna®, the first ocular gene therapy drug, the translation of retinal gene therapy from the laboratory bench to the bedside is still a work in progress that requires both sides to move in lockstep. Improvements from both basic and clinical research must come together to generate the next generation of breakthrough retinal trials; a thorough comprehension of degenerative molecular foundations must be combined with a full clinical description of the disease process. The growing availability and technological advances in studying animal disease models, as well as the development of innovative human disease models such as retinal organoids that can overcome some of the limitations of animal model study, are all encouraging developments in the field of eye translation. AAVs' versatility has enabled gene therapy, especially ocular gene therapy, to improve considerably over the past decade, bringing hope for treatment for a number of disorders previously believed to be incurable. More improvements in capsid and vector engineering, a better understanding of vector-host interactions, and clinical knowledge of vision loss illnesses will continue to provide clinicians and researchers with the tools they need to improve these novel treatments.